Adaptive pathways for medicine evaluation and unmet medical need: the access v. evidence dilemma

emea_strap_url_cmyk_rev_en_std_cent.eps MASTERThe European Medicines Agency hosted a stakeholder workshop on 8th December 2016 on adaptive pathways at its Canary Wharf London headquarters: a hot topic with around 150 delegates from healthcare, patient and consumer organisations, academia, industry and regulatory bodies.

The European Medicines Agency organised this workshop in collaboration with the European Commission to gather views and proposals from stakeholders on the adaptive pathways approach, in light of the practical experience gained during the pilot project EMA ran between March 2014 and August 2016, and to plan the next steps in the exploration of this concept.

The basis for considering adaptive pathways for evaluating medicines is to meet the challenges underlying how to resolve high unmet medical need with then aim of licensing products likely to have a major impact on patient morbidity and/or life expectancy.

The desire is to find ways to reduce unavoidable uncertainties as rapidly as possible for serious/rare debilitating and life-shortening illnesses. There are clear ethical and scientific challenges in using novel approaches to evaluate medicines without a decline in the quality of evidence on the effectiveness and safety of new treatments.

Products which might be considered for use of adaptive evaluation pathways include both conventional medicines and advanced therapy medicinal products (ATMPs). ATMPs are medicinal product which involve either a gene therapy medicinal, a somatic cell therapy or a tissue engineered product [see more on ATMPs at Directive 2001/83/EC as amended by the ATMP Regulation 1394/2007].

Adaptive pathways contribute to an expanded  toolbox for evidence generation where conventional randomized controlled trials are not-appropriate or practical. Principles include using processes to allow rapid reaction to uncertainty by iterative development based on ongoing analysis of  pathway data [“rapid cycle analysis”]. As secondary end-points are welcomed, these pathways require pre-planning across the entire product life-span, including the post-marketing paths ie active monitoring and management of on-market use.

The adaptive pathway approach is dependent on earliest stage and ongoing multi-stakeholder networking, underpinned by risk management plans.

Critical issues include

– resolving scientific trust in real world data and the need to develop mature, robust registries

– defining and prioritising unmet medical needs

– agreement on acceptable risks and benefits early access worth it (vs.

– maintaining safety standards

– ensuring that benefits outweigh risks

– avoiding unrealistic expectations

– establishing practical criteria for reimbursement, especially when secondary outcome measures form the basis for market authorization

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